Oxytocin may help autism – and may also help explain the placebo effect

Scientists at Stanford have early evidence that kids with autism benefit from treatment with oxytocin, a hormone that influences social behavior. In a trial of oxytocin nasal spray conducted in boys and girls with autism, the biggest improvements in social behavior occurred in children who had low blood oxytocin levels to begin with. The results appear todayin the Proceedings of the National Academy of Sciences.

In the past, scientists thought that children with autism might be deficient in oxytocin. Prior Stanford research showed that the truth was more complicated: kids with autism can have a range of oxytocin levels, and those with low levels of the hormone do have more social impairment. That finding coupled with the new results may help physicians target oxytocin therapy to the right patients, as our press release explains:

Our results suggest that some children with autism will benefit from oxytocin treatment more than others, and that blood oxytocin levels might be a biological sign that will allow us to predict if a child will respond maximally or not,” said lead author Karen Parker, PhD, associate professor of psychiatry and behavioral sciences. The trial, in 32 children, was relatively small and needs to be replicated, she said.

“We are finally narrowing down whom oxytocin could be beneficial for,” said Antonio Hardan, MD, professor of psychiatry and behavioral sciences and senior author of the study. “This is what precision health looks like for autism.”

Although the effect of oxytocin was modest, the results are exciting because no other medications now exist to treat any of the core features of autism, Hardan added.

The study also turned up an intriguing finding in its placebo group. As is common in psychiatric research, subjects receiving the placebo improved somewhat from the start to the end of the study, although their improvement was significantly smaller than in the oxytocin group.

Interestingly, blood oxytocin levels rose in the placebo group, meaning the placebo subjects' bodies were making more oxytocin on their own. It's possible that changes in oxytocin production may also help explain other instances of the placebo effect, Parker said, noting that the idea merits more research.

Meanwhile, to help understand the study's main finding, Parker and Hardan are awaiting the results of a larger NIH-funded trial now underway at several locations across the country.

The scenario many of us learned in school is that two X chromosomes make someone female, and an X and a Y chromosome make someone male. These are simplistic ways of thinking about what is scientifically very complex.